Fatal brain cancer tamed by gene therapy

Rats with an aggressive form of human brain cancer have been successfully treated with gene therapy that "trains" the immune system to attack tumours.

The results could pave the way for human trials early next year, say researchers who developed the system at the Cedars-Sinai Medical Center in Los Angeles.

Previous attempts to treat glioblastomas with gene therapy failed because some tumour cells survived and regrew. The new treatment overcomes this problem by permanently priming the immune system to pick off any straggler tumour cells.

A harmless virus that only infects fast-dividing cancer cells is injected directly into the brain, and used to deliver the therapeutic genes into the tumour. One gene, HSV1-TK, kills the cancer cells by activating ganciclovir, an otherwise ineffective drug administered into the rat's abdomen. Yet the key to the therapy's long-term success is a second gene, Flt3L, which summons immune "dendritic" cells from the bloodstream ...

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